Factor IX CB 2679d/ISU304

Catalyst created the improved and potent Factor IX protease, CB 2679d, also known as ISU304, for the potential treatment of hemophilia B. Catalyst believes that CB 2679d may provide individuals with hemophilia B with a viable subcutaneous prophylactic therapy and, in June 2017, the Company’s partner, ISU Abxis, initiated a Phase 1/2 proof-of-concept study in individuals with hemophilia B. The trial is an open label trial with an intravenous cross-over to subcutaneous dosing. The objective of the trial is to study the pharmacokinetics of CB 2679d, document the subcutaneous bioavailability of CB 2679d and activity levels achieved with escalating subcutaneous doses and observe steady-state levels that result from daily dosing for six days. The trial is being conducted at three centers in South Korea. The trial is expected to enroll up to 17 individuals with severe hemophilia B, and be completed in 2018.

Factor IX CB 2679d Phase 1/2 proof-of-concept study in individuals with severe hemophilia B - ongoing

The leading recombinant human Factor IX on the market for treating acute bleeding episodes in individuals with hemophilia B is delivered intravenously (due to its short half-life) and is therefore not ideal for prophylactic treatment. Extended half-life agents used for prophylaxis require intravenous dosing and have a prolonged period of low activity levels with increased risk of spontaneous bleeding. CB 2679d has shown significantly higher potency in pre-clinical studies compared with other FIX products on the market and in development.

CB 2679d has been granted orphan medicinal product designation in Europe to treat individuals with hemophilia B. Orphan medical products in Europe are required to be judged superior to currently available therapies to receive the designation and must be confirmed to be superior when marketing authorization is granted.

Catalyst has a collaboration with ISU Abxis (KOSDAQ: 086890) to advance the development of CB 2679d through a Phase 1/2 proof-of-concept study in individuals with hemophilia B. Learn more about our strategic collaboration with ISU Abxis.